Advanced Thyroid Cancer Patients in Phase I Clinical Trials: Outcome Assessment and Literature Review.

BACKGROUND

To describe the basic demographics, analyze the response and survival experience of advanced thyroid cancer subjects treated in Phase I clinical trials. We also reviewed early Phase studies using new targeted agents in thyroid cancer.

METHODS

We conducted a retrospective observational study in 32 advanced thyroid cancer patients who participated in 21 Phase I trials at our center between 2002 and 2012. Descriptive statistics and Kaplan Meier analyses were used to examine clinical outcomes and survival.

RESULTS

The median age of subjects was 57.5 years (range 21-81) at the time of study enrollment; more men (53.1%) than women were enrolled. A large number of study participants were Hispanic Americans. Nearly half (46.8%) of patients had ECOG performance status of zero, 53.1% were ECOG one and above. The most common histological subtypes were papillary (37.5%), medullary (28.1%), follicular (18.7) and anaplastic (15.6%). More than half of patients had ≥ 2 metastatic sites (62.6%). Of the 30 evaluable patients for tumor response, 2 confirmed partial responses (6.6%) were observed, whereas 17 patients had stable disease (SD) as best response. Among patients with stable disease, 10 patients (33.3%) achieved SD lasting ≥ 6 months. The median overall survival from the time of enrollment in a Phase I trial was 16.1 months. No treatment related death was observed among these patients treated with an investigational agent.

CONCLUSION

Advanced thyroid cancer patients with no standard treatment options could participate in early Phase clinical trials of novel agents.